[Federal Register Volume 76, Number 242 (Friday, December 16, 2011)]
[Proposed Rules]
[Pages 78216-78224]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2011-32204]
[[Page 78216]]
-----------------------------------------------------------------------
DEPARTMENT OF HEALTH AND HUMAN SERVICES
42 CFR Part 121
RIN 0906-AA73
Organ Procurement and Transplantation Network
AGENCY: Health Resources and Services Administration, HHS.
ACTION: Notice of proposed rulemaking.
-----------------------------------------------------------------------
SUMMARY: This notice of proposed rulemaking sets forth the Secretary's
proposal to include vascularized composite allografts, described below,
within the definition of organs covered by the rules governing the
operation of the Organ Procurement and Transplantation Network. The
Secretary further proposes a corresponding change to the definition of
human organs covered by section 301 of the National Organ Transplant
Act of 1984, as amended.
DATES: To be considered, comments on this proposed rule must be
submitted by February 14, 2012. Subject to consideration of the
comments submitted, the Department intends to publish final
regulations.
ADDRESSES: You may submit comments, identified by RIN 0906-AA73, by any
of the following methods:
Federal eRulemaking Portal: http://www.regulations.gov.
Follow the instructions for submitting comments.
Agency Web site: http://www.hrsa.gov/. Follow the
instructions for submitting comments on the Agency Web site.
Email: [email protected]. Include RIN 0906-AA73
in the subject line of the message.
Fax: (301) 594-6095.
Mail: James Bowman, M.D., Medical Director, Division of
Transplantation, Healthcare Systems Bureau, Health Resources and
Services Administration, 5600 Fishers Lane, Room 12C-06, Rockville,
Maryland 20857.
Hand Delivery/Courier: James Bowman, M.D., Medical
Director, Division of Transplantation, Healthcare Systems Bureau,
Health Resources and Services Administration, 5600 Fishers Lane, Room
12C-06, Rockville, Maryland 20857.
Instructions: All submissions received must include the agency name
and Regulatory Information Number (RIN) for this rulemaking. All
comments received will be posted without change to http://www.hrsa.gov/
, including any personal information provided. For detailed
instructions on submitting comments and additional information on the
rulemaking process, see the ``Public Participation'' heading of the
SUPPLEMENTARY INFORMATION section of this document.
Docket: For access to the docket to read background documents or
comments received, go to the Division of Transplantation, Healthcare
Systems Bureau, Health Resources and Services Administration, 5600
Fishers Lane, Room 12C-06, Rockville, Maryland 20857 weekdays (Federal
holidays excepted) between the hours of 8:30 a.m. and 5 p.m. To
schedule an appointment to view public comments, phone (301) 443-7757.
FOR FURTHER INFORMATION CONTACT: James Bowman, M.D., at the above
address; telephone number (301) 443-4861.
SUPPLEMENTARY INFORMATION: The transplant community has performed
transplants of hands and various body parts using the term composite
tissue allograft; however, for the purposes of rulemaking, the Health
Resources and Services Administration (HRSA) has defined a subset of
such body parts as vascularized composite allografts (VCA), which share
certain characteristics.
Based upon a review of VCA, the Secretary believes that VCA should
now be included within the definition of organs covered by the rules
governing the operation of the Organ Procurement and Transplantation
Network (OPTN) (hereinafter the OPTN final rule) (42 CFR part 121).
This notice sets forth the history of VCA transplants, the factors that
have persuaded the Department of the advisability of including VCA
within the authority of the regulations governing the operation of the
OPTN, the Secretary's oversight of VCA, and the anticipated
consequences of this proposal. The notice also discusses the
Department's proposal to include VCA within the definition of human
organs covered by section 301 of the National Organ Transplant Act of
1984, as amended (hereinafter section 301 of NOTA).
Public Participation
Through this notice, the Secretary seeks comments from the public
on the proposals made. Additional information on the submission of
comments and/or the rulemaking process can be obtained from the
Director, Division of Policy Review and Coordination, Health Resources
and Services Administration, 5600 Fishers Lane, Room 14A-11, Rockville,
Maryland 20857.
Background
VCA transplantation comprises transplants of a variety of body
parts (all of which contain similar characteristics, described later)
that are not currently regulated under the OPTN final rule. Perhaps the
two most notable types to date have been hand and face transplants. The
first successful hand transplant in the United States was performed in
1999 and the first face transplant in the U.S. was performed in 2008.
Worldwide, there have been over three dozen limb transplants, at least
a dozen transplants of portions of the face, and a small number of
transplants of other such anatomical parts (e.g., abdominal wall,
vascularized skeletal muscle, and digits). Accurate data about the
actual number of such transplants have been difficult to obtain because
there is no requirement for reporting these procedures in the U.S. Most
of the available information has been obtained from published news
accounts in the popular press and anecdotal reports in the medical
literature.
Although the body parts involved vary significantly, among their
shared characteristics is the fact that they are susceptible to
ischemia (damage or death from lack of blood flow) and that they need
revascularization, done through a surgical reconnection of blood
vessels to accomplish the transplant, as opposed to secondary ingrowth
of vessels. In viable vascularized transplants, immunosuppression is
necessary to prevent or treat rejection. This immunosuppression has
risks, which have been justified in patients needing organs as
presently defined in the OPTN final rule because of their lifesaving
potential. In the past, the risks of immunosuppression have inhibited
transplantation of VCA because the risks associated with the prolonged
use of immunosuppressive drugs were thought to exceed the expected
benefits of the transplants. However, the powerful impact these
transplants can have to improve the quality of life for individuals
with grievous disabilities has become increasingly apparent.
Immunosuppressive management for these transplants has also improved so
that risks associated with immunosuppression, such as cancer,
infection, or other morbidities in recipients, are lessened
considerably. (F Schuind, Hand transplantation and vascularized
composite tissue allografts in orthopaedics and traumatology,
Orthopaedics & Traumatology: Surgery & Research (2010) 96, 283-290, and
Armed Forces Institute of Regenerative Medicine Annual Report, 2009, pp
II-1 and II-62 and II-63). In recent years, the
[[Page 78217]]
Departments of Defense and Veterans Affairs have initiated substantial
funding of clinical research programs for limb and face transplantation
anticipating the reconstruction needs of wounded service members
returning from the conflicts in Iraq and Afghanistan. More than 1,000
military men and women have lost an arm or leg in these conflicts and
20 percent have lost two or more limbs. As of mid-summer 2010, it was
estimated that as many as 200 wounded troops might be eligible for face
transplantation and about 50 for hand/forearm transplants. Most of the
funding for limb and face transplantation research in the U.S.
currently comes from the Departments of Defense and Veterans Affairs
(Armed Forces Institute of Regenerative Medicine Annual Report, 2009,
pp I-1 and I-2). For these reasons, it is likely that the numbers of
VCA transplanted will increase in the future.
Human cells or tissue intended for implantation, transplantation,
infusion, or transfer into a human recipient are regulated as a human
cells, tissues, and cellular and tissue-based products or HCT/Ps. The
Food and Drug Administration (FDA) regulates HCT/Ps under 21 CFR parts
1270 and 1271. Examples of such tissues are bone, skin, corneas,
ligaments, tendons, dura mater, heart valves, hematopoietic stem/
progenitor cells derived from peripheral and cord blood, oocytes, and
semen. Face and hand allografts, and other body parts meeting the
proposed definition of VCA in this notice are currently not explicitly
excluded from the definition of HCT/Ps under FDA regulations and are
therefore subject to FDA oversight. The FDA has no statutory or
regulatory authority to mandate VCA allocation policies, direct
coordination of procurement efforts, require consistent application of
recovery and logistics processes, or establish mandatory outcomes
reporting and provide oversight of VCA transplant programs. FDA does
not regulate the transplantation of vascularized human organ
transplants such as kidney, liver, heart, lung, or pancreas. The Health
Resources Services Administration oversees the transplantation of
vascularized human organs.
Given the anticipated increase in VCA transplants, HRSA published a
Request for Information (RFI) on March 3, 2008, in the Federal Register
for the purpose of soliciting feedback from stakeholders and the public
as to whether VCA should be included within the definition of organs
covered by the OPTN final rule and/or added to the definition of human
organs covered by section 301 of NOTA. (73 FR 11420.) HRSA also sought
feedback on the optimal way to define VCA if either definitional change
was pursued.
Through the RFI, HRSA invited the public to attend a meeting on
April 4, 2008 to discuss the issues described above. The meeting was
well attended and provided a venue for discussion on VCA issues.
Participants were instructed to provide written comments and the
deadline for these comments to be received by HRSA was extended to July
2, 2008.
In response to its RFI, HRSA received 11 written comments about
whether VCA should be included within the definition of organs covered
by the OPTN final rule.
Eight of the written comments received supported including VCA
within the definition of organs covered under the OPTN final rule. Many
of these comments included similar supporting statements for OPTN
oversight. The commenters agreed that the use of the existing solid
organ transplant infrastructure would ensure rapid and equitable
placement of VCA; allow allocation of VCA over a wide geographic area;
facilitate identification of appropriate VCA donor and recipient pairs;
provide assurance that all VCA programs are following similar rules,
ensuring uniform and appropriate clinical and ethical standards on both
the donation and transplantation side; facilitate the development of
expertise and a body of knowledge that would be a valuable resource to
address questions from the government or the public, and in the
development of future policy and procedures in the field of VCA
transplantation; enhance public transparency, increasing public
acceptance of donation of VCA; and facilitate the protection of public
health and safety in the context of VCA transplantation. Commenters
also stated that the structure and goals of the OPTN are well aligned
with the types of clinical and ethical concerns raised by VCA
transplantation such as contingency treatment plan for complete face
graft loss and fear of loss of facial identity due to transfer of donor
facial characteristics (AJ Alexander et al, Arguing the Ethics of
Facial Transplantation, Arch Facial Plast Surg. 2010;12(1):60-63) and
with the types of entities that would be carrying out these activities,
e.g., organ procurement organizations (OPOs) and transplant centers.
Of the three remaining comments, two supported partial inclusion of
VCA within the OPTN final rule's definition of organs and the third
comment did not support having VCA included within the OPTN final
rule's definition of organs. Of the two comments advocating partial
inclusion of VCA under the OPTN final rule's definition of organs, one
stated that VCA should be classified as either ``life extending'' or
``not life extending.'' Life extending VCA were described as those
involving: (a) Vascularized tissue, such as the use of abdominal wall
transplanted to close a ruptured wound in a small bowel transplant
recipient; and (b) non-vascularized tissue, such as a heart valve. Not
life extending VCA were described as those involving: (c) vascularized
tissue, such as a hand transplant; and (d) non-vascularized tissue,
such as an anterior cruci, bone, or nerve grafts. The commenter
supported including ``life extending'' VCA (a and b in the above
examples) under the definition of organs under the OPTN final rule.
According to this commenter, all life-saving VCA should follow the same
rigorous testing and screening of donors and the procurement that is
currently conducted by the OPTN contractor for organs currently covered
under the OPTN final rule. However, the responder strongly opposed
regulating ``not life extending'' VCA (c and d in the above examples),
which are not conventional organ grafts, under the OPTN final rule. The
responder suggested that although the OPTN should regulate control of
distribution of the grafts, these two ``not life extending'' types (c
and d) should be subject to less oversight. The commenter recommended
new oversight legislation that would not hamper the innovation and
utilization of these novel types of VCA. The Secretary wishes to make
clear that certain of the body parts discussed by this commenter (e.g.,
non-vascularized tissues, such as heart valves and anterior cruci,
bone, or nerve grafts, regardless of whether they would be considered
life-saving or life-enhancing) are regulated by the Food and Drug
Administration (FDA) as HCT/Ps. (21 CFR part 1271).
The second comment supported limited oversight of VCA by the OPTN
at this time. The commenter supported OPTN oversight with respect to
designation of VCA transplant programs, data submission regarding
transplant procedures, and donor screening. However, the commenter does
not support allocation policies for VCA at this time due to the unknown
clinical demand and overall future of these transplants. As noted
above, clinical demand for VCA transplantation appears to be increasing
now that immunosuppression protocols have proven safer and support for
[[Page 78218]]
military and veterans VCA transplantation programs continues to expand.
The issues concerning allocation, recipient safety, and outcomes
reporting are similar for VCA and for organs currently under the OPTN's
auspices. The VCA transplant community has clearly indicated its
support for Federal oversight of VCA as organs through the OPTN in a
letter of request from the Association of Organ Procurement
Organizations to the Assistant Secretary of Health (December 9, 2010)
and a publication of recommendations by the American Society of
Transplant Surgeons in 2011 (Implementation of Vascularized Composite
Allografts in the United States, American Journal of Transplantation
(2011) 11:13-17),
The third comment did not support including VCA within the OPTN
final rule's definition of organs. The comment stated that VCA do not
fit as organs under HRSA oversight due to differences between solid
organs procured for transplantation with the intent to save lives and
VCA that are not used in life-saving applications. It also stated that
the regulations that govern organ donation and transplantation are
designed to maximize donation and to provide organs to as many waiting-
list recipients as possible to avoid death due to their medical
illness. According to the commenter, VCA recipients should not be
subject to the same risks of donor transmissible diseases as recipients
of traditional solid organs (e.g., heart, lung, liver, and kidney). The
commenter suggests that human-derived graft materials which enhance
lives can be designated by Federal regulations under oversight of FDA
as either an HCT/P, a biologic, or a medical device. However, both
traditional organs and VCA originate from the same pool of potential
donors and therefore subject all of these transplant recipients to
similar risks of donor transmissible diseases. As described elsewhere,
VCA share anatomic, clinical, allocation-logistical characteristics
more closely related to those of traditional organs than biologics or
medical devices. Therefore, in the Secretary's view, the appropriate
way to distinguish those body parts that should be regulated as organs
under the OPTN final rule and those that should not be similarly
defined is based upon the properties of the body parts themselves
rather than whether the intent is considered life-saving or life-
enhancing. The OPTN final rule does allow some flexibility specific to
each organ such that the OPTN may develop distinct organ-specific
policies tailored to the circumstances, including risk of donor
transmissible disease by transplanting particular organs.
The Secretary has considered comments that VCA such as hand
transplants are not life saving and therefore are different from organs
presently under HRSA and OPTN oversight. The Secretary does not agree
with a direct demarcation between life-saving organ transplants and
life-enhancing organ transplants for the purposes of defining organs
under the OPTN final rule. The kidney has always been included in the
list of organs governed by NOTA, the OPTN final rule, and the OPTN.
Until additional life years provided by a kidney transplant (as opposed
to continuing dialysis) were demonstrated, a kidney transplant was not
understood to be life-saving. This fact did not dissuade the Congress
from determining that kidneys should be subject to oversight under NOTA
at its original enactment in 1984. As a result, kidney transplants are
under the purview of the OPTN final rule and the OPTN (and subject to
oversight by HRSA). Moreover, instead of adopting an all or nothing
approach (life-extending versus life-enhancing), it may be better to
understand improvements to the quality of life and increases to the
length of life as coexisting on a spectrum of benefits. Hand and face
transplants, and transplants of other body parts qualifying as VCA
under the definition proposed here, may prove to be more powerful in
improving a recipient's quality and extension of life than previously
understood. In the Secretary's view, the appropriate way to distinguish
between those VCA that should be regulated as organs under the OPTN
final rule and body parts that should not be similarly defined is based
upon the properties of the body parts themselves, rather than their
potential impact upon the lives of their recipients.
Upon consideration of the comments received, and for the reasons
described below, the Secretary now proposes that transplants of VCA be
regulated under the OPTN final rule and governed by section 301 of
NOTA.
Adding VCA to the Definition of Organs Covered by the OPTN Final Rule
Through this notice, the Department proposes adding VCA to the
definition of organs included in the OPTN final rule, codified at 42
CFR 121.2, through rulemaking. When it enacted NOTA in 1984, Congress
included a definition of the term organ and authorized the Secretary to
expand this definition by regulation. The Secretary has previously
exercised this authority and expanded the statutory definition of
organ. Currently, the OPTN final rule defines covered organs as ``a
human kidney, liver, heart, lung, or pancreas, or intestine (including
the esophagus, stomach, small and/or large intestine, or any portion of
the gastrointestinal tract). Blood vessels recovered from an organ
donor during the recovery of such organ(s) are considered part of an
organ with which they are procured for purposes of this part if the
vessels are intended for use in organ transplantation and labeled `For
use in organ transplantation only.'''
One of the major reasons NOTA was enacted and affirmed by several
amendments was to establish an organ allocation system that functions
equitably on a nationwide basis with provisions for outcomes reporting
and evaluation. Prior to the enactment of NOTA, deceased donor organs
were allocated regionally, based on relationships between transplant
programs and donor hospitals. Congress recognized the need to allocate
this national resource on a national and equitable basis. To ensure
equitable access for those awaiting VCA transplantation, there is a
need to provide for consistency in allocation processes and reliable
outcomes reporting on a nationwide basis. Appropriate Federal oversight
of a national allocation system can increase safety of such transplants
and provides equitable and consistent national access to such
transplants while also conveying to the public that donation for such
purpose will serve an essential medical need. The FDA does not have
statutory authority to provide oversight of VCA allocation, outcomes
reporting, or promotion of donation. The Secretary believes that the
rationale for a national system of organ allocation and outcomes
reporting underlying NOTA applies to VCA.
Once a body part is defined as an organ under the OPTN final rule,
such body parts are excluded from the coverage of FDA regulations
governing HCT/Ps. In addition, transplants involving body parts defined
as organs under the OPTN final rule are subject to the requirements of
the OPTN final rule. For example, entities performing transplants with
covered organs must receive designation as an organ-specific designated
transplant program (in this case, a designation as a VCA-specific
transplant program) within an OPTN member institution. Members must
comply with data submission requirements of the OPTN final rule and are
subject to oversight by the OPTN contractor for compliance with OPTN
policies, OPTN bylaws, and the OPTN
[[Page 78219]]
final rule. Members may be subject to Federal enforcement actions for
violations of Federal regulations or enforceable policies (those
approved by the Secretary of Health and Human Services) or for actions
or inactions that indicate a risk to health of patients or to the
public safety. Finally, OPTN members can be subject to OPTN sanctions
for violating OPTN bylaws and non-enforceable OPTN policies (e.g.,
being declared a member not in good standing).
As previously discussed and also explained in ``Statement of Need''
within the ``Impact of the New Rule'' section (below), the Secretary
believes that oversight of the VCA transplants is necessary to ensure
transplant recipient safety and to provide a consistent allocation
process nationwide that will ensure equitable access to those waiting
for VCA transplantation., to collect data on VCA transplant outcomes,
and to maintain the public trust in the integrity of the VCA donation,
recovery and transplant processes. Because of the clinical,
procurement, logistical, allocation, and outcomes reporting
similarities between VCA and organs currently under the OPTN's
auspices, the Secretary believes that HRSA is the appropriate HHS
agency to assure Federal oversight over VCA transplantation. HRSA
oversees transplantation of vascularized human organs through the OPTN,
which sets policies related to the procurement, transplantation,
allocation, and outcomes reporting of human organs. The OPTN serves the
critical role of matching donor organs to potential recipients on a
national basis. The issues concerning allocation and recipient safety
are similar for VCA and for organs currently under the OPTN's auspices.
Additionally, the membership of the OPTN, which is charged with
developing policies consistent with the OPTN final rule, includes
professionals with expertise in the field. Therefore, the Secretary
believes that the OPTN, with HRSA's oversight, will be able to
effectively address issues involving the regulation of the emerging
field of VCA transplantation.
If VCA are included within the OPTN final rule's definition of
organs, transplants involving VCA will be subject to the requirements
of the OPTN final rule. For example, an entity performing VCA
transplants would have to receive designation as a VCA-designated
transplant program within an OPTN member institution. In addition, OPTN
members would be required to comply with the OPTN final rule's data
submission requirements with respect to the transplants performed.
Thus, the OPTN would need to devise certain policies with respect to
VCA, including allocation policies meeting the requirements set forth
in the OPTN final rule. Finally, OPTN members would be subject to
oversight by the OPTN contractor for compliance with OPTN policies
extending to VCA (e.g., those concerning donor screening and
allocation), and could be subject to enforcement actions for violations
of such policies.
Even so, the OPTN final rule does allow some flexibility specific
to each organ. The OPTN sometimes fashions distinct organ-specific
policies tailored to the circumstances of transplanting particular
organs. For example, the training of professionals working for
designated programs may vary by organ and OPTN policies with respect to
disease transmission protocols and testing may diverge based on
circumstances relating to particular organs. Likewise, the particular
characteristics of and circumstances surrounding different types of
organs lead to different OPTN allocation policies.
In addition, if VCA are added as covered organs under the OPTN
final rule as proposed here, the Secretary will continue to exercise
oversight over proposed and final OPTN policies with respect to VCA,
consistent with the authority of the Secretary under 42 CFR 121.4.
Given the relatively small numbers of other VCA transplanted at this
time, the Secretary does not expect that the OPTN would develop
allocation policies for all VCA within a short time frame if VCA are
added to the OPTN final rule's definition of organs. We expect that the
OPTN will initially create policies addressing hands and faces as these
two VCA have been the most frequently performed VCA transplant
procedures in the U.S. and are the subject of extensive ongoing
clinical research programs by the Departments of Defense and Veterans
Affairs. We expect that the OPTN will wait to develop allocation
policies for other VCA until the field has more clinically evolved.
Given the Secretary's substantial interest in VCA policy and
involvement in the operations of the OPTN, the Secretary will be
notified of proposals to develop policies for other VCA as they are
addressed in the future.
The nature of the regulatory framework governing the operation of
the OPTN underlies the importance of including VCA within the
definition of organs covered by the OPTN final rule. Under the OPTN
final rule, the OPTN must submit proposed policies for review and
approval by the Secretary (42 CFR 121.4). Upon consideration of public
comments on proposed policies that are considered significant, the
Secretary will determine whether to make such proposed policies
enforceable in accordance with section 121.10 of the OPTN final rule.
The Secretary may direct the OPTN to develop individual policies for
specific body components that are defined as VCA in addition to OPTN
policies that apply to all VCA. Any transplant hospital that fails to
comply with any policy approved as enforceable by the Secretary under
this process may be subject to the enforcement sanctions delineated in
section 121.10 of the OPTN final rule, including possible termination
from the Medicare and Medicaid programs.
The Secretary has the following additional authorities provided by
the OPTN final rule (42 CFR 121.4(b)(2)), which she may exercise in the
case of policies extending to VCA: The Secretary may require the OPTN
Board of Directors to provide to the Secretary, at least 60 days prior
to their proposed implementation, proposed policies on matters that the
Secretary directs. The Secretary will refer significant proposed
policies to the Advisory Committee on Organ Transplantation (ACOT)
established under 42 CFR 121.12, and publish them in the Federal
Register for public comment. This is in addition to the public comment
process that is engaged in by the OPTN.
The Secretary also may seek the advice of the ACOT on other
proposed policies and publish them in the Federal Register for public
comment.
The Secretary will determine whether proposed policies are
consistent with NOTA and the OPTN final rule, taking into account the
views of the ACOT and public comments. Based on this review, the
Secretary may provide comments to the OPTN.
If the Secretary concludes that a proposed policy is inconsistent
with NOTA or the OPTN final rule, the Secretary may direct the OPTN to
revise the proposed policy consistent with the Secretary's direction.
If the OPTN does not revise the proposed policy in a timely manner, or
if the Secretary concludes that the proposed revision is inconsistent
with NOTA or the OPTN final rule, the Secretary may take such other
action as the Secretary determines appropriate, but only after
additional consultation with the ACOT on the proposed action.
Also, the Secretary has the authority under the OPTN Final Rule (42
CFR 121.4(a)(6)) to require the OPTN to develop policies on such
matters as the Secretary directs.
[[Page 78220]]
The Secretary is legally obliged, as part of her responsibilities
in administering the Medicare and Medicaid programs, to require
hospitals that transplant organs to comply with the rules and
requirements of the OPTN as a condition of their participation in
Medicare and Medicaid. (42 U.S.C. 1320b-8(a)(1)(B).) Because VCA are
not included within the OPTN final rule's definition of organs, the
Secretary could not currently make any VCA allocation policy
enforceable. If VCA are added as covered organs under the OPTN final
rule as proposed here, the Secretary could take appropriate enforcement
actions against an OPO or transplant hospital for failing to comply
with the OPTN's VCA retrieval and allocation policy, if such a policy
has been approved as enforceable by the Secretary under the process
outlined above. If VCA are defined as organs under the OPTN final rule,
then this will ensure that VCA organ allocation, whether pertaining to
isolated VCA transplants or combined/multi-organ transplants, is
consistent with OPTN final rule's goals, including that of an equitable
national system for organ allocation, to be consistent with NOTA.
Even if OPTN policies pertaining to VCA transplantation do not
become enforceable, all institutions performing VCA transplantation
would be required to comply with the provisions of the OPTN final rule
(including the requirement that such institutions become members of the
OPTN). Further, such institutions could be subject to sanctions by the
OPTN for failure to comply with allocation and other OPTN policies. For
example, a member may be named a member not in good standing by the
OPTN for failing to comply with such a policy.
As the field of VCA transplantation evolves, it will become more
critical that VCA organ allocation keeps pace with advances in the
field; that the field be subject to appropriate Federal oversight; that
policy developments include performance indicators to assess whether
the goals of an equitable transplant system are being achieved; that
the Secretary have the authority to make those policies enforceable;
and that patients and physicians have timely access to accurate data
that will assist them in making decisions regarding VCA
transplantation. Upon consideration of the foregoing factors, and to
achieve the most equitable and medically effective use of donated
organs, the Secretary proposes that VCA should explicitly be added to
the definition of organs covered by the OPTN final rule. The Secretary
seeks comments on this proposal.
Defining Vascularized Composite Allografts
At the time of the RFI, and to assist the Secretary in adding VCA
to the definition of organs covered by the OPTN final rule and/or to
the definition of human organs governed by section 301 of NOTA, HRSA
sought feedback from stakeholders and from the public as to how such
allografts should be defined. HRSA identified two potential approaches:
(1) A broad regulatory definition describing the common features of VCA
without listing covered body parts; or (2) a definition listing body
parts that would qualify as VCA.
The Secretary has elected to propose the first approach, a broad
regulatory definition that describes the features of the allografts
without listing particular body parts. Under this approach, the
definition would extend to transplants of particular body parts that
are not known to have been performed clinically to date, or even to
body parts whose transplantation has not yet been envisioned. The
Secretary is proposing which elements should be included in the
definition of VCA to be sufficiently broad to cover the universe of
intended body parts, but narrow enough to put the public on notice as
to which parts meet the regulatory definitions of organs.
The Secretary proposes that for a body part to be defined as a VCA,
it must have all the following characteristics: a body part (1) That is
vascularized and requires blood flow by surgical connection of blood
vessels to function after transplantation; (2) containing multiple
tissue types; (3) recovered from a human donor as an anatomical/
structural unit; (4) transplanted into a human recipient as an
anatomical/structural unit; (5) minimally manipulated, (processing that
does not alter the original relevant characteristics of the organ
relating to the organ's utility for reconstruction, repair, or
replacement--examples of minimal manipulation include cutting,
grinding, and shaping of a VCA); (6) for homologous use, (the
replacement or supplementation of a recipient's organ with an organ
that performs the same basic function or functions in the recipient as
in the donor), (e.g., a hand from the donor is to be used as a hand in
the recipient); (7) not combined with another article such as a device;
(8) susceptible to ischemia and, therefore, only stored temporarily
(e.g., cold storage in preservation medium and intended for
implantation into a recipient within hours of the recovery) and not
cryopreserved; and (9) susceptible to allograft rejection, requiring
immunosuppression that may increase infectious disease risk to the
recipient. This proposed definition is intended to explain to the
public which body parts would be covered presently, while allowing
other body parts that are transplanted to be covered as the field of
VCA transplantation advances. A non-exclusive list of body parts that
would meet the proposed definition for VCA here would include faces,
hands, fingers, toes, larynges, and abdominal walls. Periodically, HRSA
may publish an updated list of VCA in the Federal Register. In
addition, through this definition, the Secretary intends to distinguish
those body parts she proposes to define as organs under the OPTN final
rule from other body parts that are regulated as HCT/Ps under FDA's
regulatory authority.
Under a second alternative, the Secretary could have proposed a
definition that lists specific transplantable body parts to be added to
the definition of organs (e.g., face, hand, etc.). The Secretary finds
this unnecessary since the general set of nine characteristics provide
clear identification of such body parts. Moreover, definition by an
explicit list would likely exclude certain body parts for which
transplantation might be possible, but not done to date (either in the
United States or internationally). The Secretary is proposing the more
descriptive definition to avoid the need of amending the regulatory
definition to extend its reach to new types of transplantation that
emerge in the future.
HRSA received no negative feedback in response to its request for
information on adopting this first approach or on the criteria
discussed in the request for information (other than the comment
distinguishing between those grafts that are lifesaving and those that
are life enhancing). Most of the commenters supporting the inclusion of
VCA in the definition of organs covered by the OPTN final rule would
defer to the physicians and surgeons involved to determine the optimal
way to define VCA. Given that Congress authorized the Department to
modify the definition of covered organs through rulemaking, it would
not be permissible to allow transplant surgeons and physicians (or
others participating in the OPTN), on their own, to define VCA for the
purposes of the final rule. However, the Secretary seeks feedback from
the transplant community on the definition of VCA proposed here.
Additionally, body parts allocated as VCA are intended to be used
``intact'' as a VCA until the transplant center receiving the VCA
determines that a
[[Page 78221]]
portion of the VCA is not needed for transplantation of the remainder
of the VCA. If portions of a VCA are not used in connection with the
same transplant (for example, left over bone or tendons from a limb
allocated as a VCA), such body parts cannot be used for other purposes
including transplantation in a different anatomical location in the
recipient who received the VCA or in a different recipient. Disposition
of such remnants would be subject to OPTN policies.
Because the Secretary is proposing a definition that does not
identify specific VCA by name, the Secretary proposes amending 42 CFR
121.4(e) to make clear that the OPTN must identify the specific body
parts covered by any OPTN policy specific to VCA. The purpose of this
proposal is to ensure that all OPTN members and stakeholders understand
the body parts covered by OPTN policies specific to VCA. Under this
proposal, any OPTN policy that applies broadly to organs would apply to
all body parts meeting the proposed definition for VCA unless otherwise
provided for.
State registries for organ and tissue donors generally provide the
option to select organs, tissues, both, or neither. In the future we
anticipate that states will likely further distinguish VCAs and will
continue to permit individuals to select what they wish to donate. The
potential impact of including VCA in the definition of organs on organ
donation efforts, including the number of deceased donor organs that
may become available, has not been explored. Therefore, the Secretary
is seeking public comment on what impact this proposed expanded
definition of organs may have on efforts to increase participation in
deceased organ donor registries, signing organ donor cards, and general
willingness of individuals to agree to be deceased organ donors.
Including VCA Within the Definition of Human Organs Covered by Section
301 of NOTA
The Secretary further proposes including VCA within the definition
of human organs, as covered by section 301 of NOTA, which prohibits the
purchase or sale of human organs for human transplantation. This
criminal prohibition provides in part that ``[i]t shall be unlawful for
any person to knowingly acquire, receive, or otherwise transfer any
human organ for valuable consideration for use in human transplantation
if the transfer affects interstate commerce. The preceding sentence
does not apply with respect to human organ paired donation.'' (42
U.S.C. 274e(a).) Section 301 of NOTA defines the term ``human organ''
to mean ``the human (including fetal) kidney, liver, heart, lung,
pancreas, bone marrow, cornea, eye, bone, and skin or any subpart
thereof and any other human organ (or any subpart thereof, including
that derived from a fetus) specified by the Secretary of Health and
Human Services by regulation.'' (42 U.S.C. 274e(c)(1).)
As set forth by statute, Congress authorized the Secretary to add
additional organs to the definition of human organ covered by section
301 through rulemaking to include the transplantation of additional
human organs within section 301's prohibition. The Secretary has
previously exercised this authority. Adding VCA to this definition of
human organs will subject persons violating its terms to VCA to
criminal penalties.
Through this notice, the Secretary proposes to add VCA to the list
of human organs covered by section 301 of NOTA. The Secretary proposes
modifying 42 CFR 121.13, which includes the definition of human organs
covered by section 301 of NOTA, to include VCA (as defined in the
proposed amendment to section 121.2 of the OPTN final rule). Subparts
are being added to this definition to conform with Public Law 100-607,
which added subparts of covered human organs to the statutory
definition of human organs governed by section 301 of NOTA.
Economic and Regulatory Impact
Executive Order 12866 directs agencies to assess all costs and
benefits of available regulatory alternatives and, when rulemaking is
necessary, to select regulatory approaches that provide the greatest
net benefits (including potential economic, environmental, public
health, safety, distributive and equity effects). In addition, under
the Regulatory Flexibility Act, if a rule has a significant economic
effect on a substantial number of small entities the Secretary must
specifically consider the economic effect of a rule on small entities
and analyze regulatory options that could lessen the impact of the
rule.
Executive Order 12866 requires that all regulations reflect
consideration of alternatives, costs, benefits, incentives, equity, and
available information. Regulations must meet certain standards, such as
avoiding an unnecessary burden. Regulations that are significant
because of cost, adverse effects on the economy, inconsistency with
other agency actions, effects on the budget, or novel legal or policy
issues, require special analysis.
The Secretary has determined that minimal resources are required to
implement the requirements in this rule because organizations involved
(e.g., OPOs and transplant hospitals) already implement related
requirements for other organs in the OPTN rule (42 CFR 121.2).
Therefore, in accordance with the Regulatory Flexibility Act of 1980
(RFA), and the Small Business Regulatory Enforcement Act of 1996, which
amended the RFA, the Secretary certifies that this rule will not have a
significant impact on a substantial number of small entities.
The Secretary also has determined that this proposed rule does not
meet the criteria for a major rule as defined by Executive Order 12866
and would have no major effect on the economy or Federal expenditures.
We have determined that the proposed rule is not a major rule within
the meaning of the statute providing for Congressional Review of Agency
Rulemaking, 5 U.S.C. 801. Similarly, it will not have effects on State,
local, and tribal governments or on the private sector such as to
require consultation under the Unfunded Mandates Reform Act of 1995.
The provisions of this rule will not affect the following elements
of family well-being: Family safety, family stability, marital
commitment; parental rights in the education, nurture, and supervision
of their children; family functioning, disposable income, or poverty;
or the behavior and personal responsibility of youth, as determined
under section 654(c) of the Treasury and General Government
Appropriations Act of 1999.
As stated above, this proposed rule would modify the regulations
governing the OPTN and section 301 of NOTA based on legal authority.
Impact of the New Rule
Statement of Need
The field of VCA transplantation has advanced from the first hand
transplant in the U.S. in 1999 to the point that there are now more
than a dozen VCA transplant centers extending from coast to coast
involving hand, face, abdominal wall, larynx, and possibly other body
parts. The Departments of Defense and Veterans Affairs have invested
hundreds of millions of dollars in clinical VCA transplantation
research programs for the benefit of wounded warriors returning from
the Iraq and Afghanistan conflicts with extensive debilitating injuries
of the face and multiple extremities. Although the current activity
level is less than a dozen transplants a year in the U.S., the VCA
transplant community has begun to encounter the expansion problems
faced
[[Page 78222]]
in the early days of organ transplantation with ensuring equitable
access for patients to VCA, uniform allocation policies across the
U.S., coordination of procurement efforts, consistent application of
recovery and logistics processes, and monitoring patient safety with
appropriate outcomes reporting and oversight of transplant programs.
VCA transplantation consists of surgical transplants of a variety
of body parts that currently do not fall within the current regulatory
definition of ``organ'' covered by the rules governing the operation of
the OPTN. Face and hand allografts, and other body parts meeting the
definition of VCA in this notice, currently are subject to FDA
oversight under 21 CFR parts 1270 and 1271. VCA, like organs, differ
from tissues in that they must be transplanted within hours (not months
or years), recipients require immunosuppression drugs to prevent or
treat rejection, and the allocation process requires specific genetic
and clinical matching between donor and recipient.
The FDA has no statutory or regulatory authority to mandate
allocation policies, direct coordination of procurement efforts,
require consistent application of recovery and logistics processes, or
establish mandatory outcomes reporting and provide oversight of VCA
transplant programs. In short, the FDA's authority for regulation of
tissues like VCA stops at the hospital door. Only the OPTN, under HRSA
oversight, can provide reliable consistent and mandatory mechanisms and
infrastructure to address these problems facing the VCA transplant
community. Recognizing the need for such efforts to continue to advance
the field of VCA transplantation, the VCA transplant community
specifically requested the Secretary of the Department of Health and
Human Services to provide the necessary regulatory change to define
those body parts meeting the definition of VCA in this notice as
``organs'' under the OPTN Final Rule so that VCA transplantation can
continue to progress in an appropriate manner that will best serve the
patients in need of such allografts.
This proposed rule would have the effect of including VCA within
the ambit of the regulations governing the operation of the OPTN, and
would include transplanted human VCA within the prohibition set forth
at section 301 of NOTA. If implemented, the proposals set forth in this
rule would authorize the Secretary to take enforcement actions against
entities violating OPTN policies pertaining to the transplantation of
VCA once such policies are approved as enforceable by the Secretary.
Even if the Secretary does not approve such policies as enforceable,
OPTN members will be subject to enforcement actions by the OPTN for
violations of OPTN policies extending to VCA. If this rule is
promulgated, OPTN members will be required to comply with requirements
set forth in the OPTN final rule, including those pertaining to data
submission, as applied to VCA. Finally, if this proposal is
implemented, individuals violating section 301 of NOTA with respect to
VCA transplants would be subject to criminal penalties.
If this rule takes effect, transplant centers that perform VCA
transplantation would be required to take the necessary steps to ensure
that VCA transplant programs are in compliance with any policies
enacted by the OPTN specific to designated VCA allografts (e.g. hand,
face). Such policies typically specify the clinical submission
requirements for candidate registration on the waiting list, clinical
information of the transplant procedure, follow up reporting on graft
and patient outcomes, and reporting of potential donor disease
transmission events.
Paperwork Reduction Act of 1995
The amendments proposed in this notice of proposed rulemaking
contain information collection activities that are very similar to, and
based on the data collection requirements in, the OPTN final rule
approved by the Office of Management and Budget (OMB No. 0915-0157 and
OMB No. 0915-0184). Membership in the OPTN is determined by submission
of application materials to the OPTN demonstrating that the applicant
meets all required criteria for membership and will agree to comply
with all applicable provisions of the National Organ Transplant Act, as
amended, 42 U.S.C. 273 et seq. Section 1138 of the Social Security Act,
as amended, 42 U.S.C. 1320b-8 requires that hospitals in which
transplants are performed be members of, and abide by, the rules and
requirements (as approved by the Secretary of the HHS) of the OPTN as a
condition of participation in Medicare and Medicaid for the hospital.
Section 1138 contains a similar provision for the organ procurement
organizations (OPOs) and makes membership in the OPTN and compliance
with its operating rules and requirements (as approved by the Secretary
of the HHS), including those relating to data collection, mandatory for
all transplant programs and OPOs. The information is used predominantly
to match donor organs with recipients, to monitor compliance of member
organizations with OPTN policies and requirements to guide organ
allocation policy development, and to report periodically on the
clinical and scientific status of organ donation and transplantation in
this country.
The currently-approved data collection includes worksheets and
burden for organs and describes respondents as non-profit institutions
and small organizations, which would be the same for this proposed
rule. The title, description, and respondent description of all
information collections relating to VCA are shown below with similar
estimates of annual reporting and record keeping burden as with other
organs previously approved in the OPTN final rule.
Currently there are approximately 10 hand, 2 face, and 1 abdominal
wall transplant programs in the U.S., although only 7 have actually
performed a clinical transplant operation to date. Since the current
rate of VCA transplants is less than 10 a year (hand) and less than 1 a
year (face and abdominal wall), for reporting burden calculations
(below) we have projected a total of 20 VCA transplant programs each
registering 2 candidates a year to the waiting list and each program
performing 1 transplant procedure a year. The data burden calculation
in the table below assumes that data associated with entering deceased
donor information is already accounted in the current OMB approved data
collection forms. Specifically, it is reasonable to assume that any
donor that would be considered as a VCA donor is also considered to be
a donor for other organs covered by this rule. The hourly rate used for
calculation of total burden cost to respondents is the average hourly
wage for a transplant data coordinator ($26.00). This rate reflects the
median annual salary and benefits for a Data Control Clerk II
(www.salary.com) The total annual respondent burden hours (202)
represents 10.1 hours ($262.60) per respondent.
Title: Organ Procurement and Transplantation Network.
Description: Information will be collected from transplant
hospitals, organ procurement organizations, and histocompatibility
laboratories predominantly for the purpose of matching donor VCA with
potential recipients, monitoring compliance of member organizations
with system rules, conducting statistical analyses, and developing
policies relating to organ procurement and transplantation.
The practical utility of the data collection is further enhanced by
[[Page 78223]]
requirements that the OPTN must report a variety of data to the
Secretary, including data on performance by organ and status category,
including program-specific data, OPO specific data, data by program
size, and data aggregated by organ procurement area, OPTN region, the
nation as a whole, and other geographic areas (42 CFR 121.8(c)(3)). The
OPTN must also transmit proposed allocation policies and performance
indicators which will be used to assess the likely effects of policy
changes and to ensure that the proposed policies are consistent with
the OPTN final rule.
The OPTN and Scientific Registry must make available to the public
timely and accurate information concerning the performance of
transplant programs, and must respond to requests from the public for
data needed for bona fide research or analysis purposes or to assess
the performance of the OPTN or Scientific Registry, to assess
individual transplant programs, or for other purposes (42 CFR
121.11(b)(1)(C)).
The OPTN must provide to each member OPO and transplant hospital
the plans and procedures for reviewing applications and for monitoring
compliance with these rules and OPTN policies. The OPTN must also
report to the Secretary on OPOs and transplant hospitals that may not
be in compliance with these rules or OPTN policies, and on their
progress toward compliance.
The OPTN and Scientific Registry are required to maintain and
manage the information on candidates, donors and recipients.
Description of Respondents: Non-profit institutions and small
organizations. The estimated annual reporting burden is as follows:
--------------------------------------------------------------------------------------------------------------------------------------------------------
Total burden
Section Form Number of Responses per Total Average hours hours/ cost
respondents respondent responses per response ($)
--------------------------------------------------------------------------------------------------------------------------------------------------------
121.6(c).......................... Establishing Criteria for VCA 20 1 20 0.5 10
Acceptance. $260.00
121.7(b)(4)....................... Reasons for Refusal................. 20 50 1000 0.1 100
$2,600.00
121.9(b).......................... Designated Transplant Program 20 1 20 2.0 40
Requirements. $1,040.00
121.11(b)(2)...................... Recipient Histo-compatibility....... 20 1 20 0.2 4
$104.00
VCA Candidate Registration.......... 20 2 40 0.5 20
$520.00
121.11(b)(2)...................... VCA Recipient Registration.......... 20 1 20 0.75 15
$390.00
121.11(b)(2)...................... VCA Follow-Up....................... 20 1 20 0.65 13
$338.00
Total......................... .................................... 20 .............. 1,140 0.18 202
$5,252.00
--------------------------------------------------------------------------------------------------------------------------------------------------------
List of Subjects in 42 CFR Part 121
Health care, Hospitals, Organ transplantation, Reporting and
recordkeeping requirements.
Dated: August 18, 2011.
Mary Wakefield,
Administrator, Health Resources and Services Administration.
Approved: September 7, 2011.
Kathleen Sebelius,
Secretary.
Accordingly, 42 CFR part 121 is proposed to be amended as set forth
below:
PART 121--ORGAN PROCUREMENT AND TRANSPLANTATION NETWORK
1. The authority citation for part 121 continues to read as
follows:
Authority: Sections 215, 371-376 of the Public Health Service
Act (42 U.S.C. 216, 273-274d); sections 1102, 1106, 1138 and 1871 of
the Social Security Act (42 U.S.C. 1302, 1306, 1320b-8 and 1395hh);
and section 301 of the National Organ Transplant Act, as amended (42
U.S.C. 274e).
Sec. 121.2 [Amended]
2. Amend Sec. 121.2 to revise definition for Organ and add
definition for Vascularized composite allograft to read as follows:
* * * * *
Organ means a human kidney, liver, heart, lung, pancreas, intestine
(including the esophagus, stomach, small and/or large intestine, or any
portion of the gastrointestinal tract), or vascularized composite
allograft (defined in this section). Blood vessels recovered from an
organ donor during the recovery of such organ(s) are considered part of
an organ with which they are procured for purposes of this part if the
vessels are intended for use in organ transplantation and labeled ``For
use in organ transplantation only.''
* * * * *
Vascularized composite allograft means a body part:
(1) That is vascularized and requires blood flow by surgical
connection of blood vessels to function after transplantation;
(2) Containing multiple tissue types;
(3) Recovered from a human donor as an anatomical/structural unit;
(4) Transplanted into a human recipient as an anatomical/structural
unit;
(5) Minimally manipulated, (processing that does not alter the
original relevant characteristics of the organ relating to the organ's
utility for reconstruction, repair, or replacement);
(6) For homologous use, (the replacement or supplementation of a
recipient's organ with an organ that performs the same basic function
or functions in the recipient as in the donor);
(7) Not combined with another article such as a device;
(8) Susceptible to ischemia and, therefore, only stored temporarily
and not cryopreserved; and
(9) Susceptible to allograft rejection, requiring immunosuppression
that may increase infectious disease risk to the recipient.
3. In Sec. 121.4, add paragraph (e)(3) to read as follows:
Sec. 121.4 OPTN policies: Secretarial review and appeals.
* * * * *
(e) * * *
(3) identify all covered body parts in any policies specific to
vascularized composite allografts, defined in Sec. 121.2.
4. Revise Sec. 121.13 to read as follows:
[[Page 78224]]
Sec. 121.13 Definition of Human Organ Under section 301 of the
National Organ Transplant Act of 1984, as amended.
Human organ, as covered by section 301 of the National Organ
Transplant Act of 1984, as amended, means the human (including fetal)
kidney, liver, heart, lung, pancreas, bone marrow, cornea, eye, bone,
skin, intestine (including the esophagus, stomach, small and/or large
intestine, or any portion of the gastrointestinal tract), or any
vascularized composite allograft defined in Sec. 121.2. It also means
any subpart thereof, including that derived from a fetus.
[FR Doc. 2011-32204 Filed 12-15-11; 8:45 am]
BILLING CODE 4165-15-P